WELCOME TO THE ALS ONE
2020 ALS RESEARCH SYMPOSIUM November 4th & 5th - Together from afar. A conference bringing together researchers and scientists who are working on therapeutic options, and ultimately a cure, for Amyotrophic Lateral Sclerosis (ALS).
With gratitude to this year's Symosium sponsors:
"THE FUTURE IS NOT SOME PLACE WE ARE GOING TO, BUT ONE WE ARE CREATING. THE PATHS TO IT ARE NOT FOUND, BUT MADE; AND THE ACTIVITY OF MAKING THEM CHANGES BOTH THE MAKER AND THE DESTINATION"— PETER ELLYARD
PROGRAM
PAGE
CONTENT
3
ABOUT ALS ONE
4-7
HOSTS & COMMITTEE
8-10
AGENDAS: DAY 1 & 2
11-16
KEYNOTE SPEAKER BIOGRAPHIES
17-23
INDUSTRY SPEAKER BIOGRAPHIES
24-27
ROUNDTABLE SPEAKER BIOGRAPHIES
28-34
SPONSORS
35-36
NOTES
37
ALS ONE'S DRIVE TO END ALS
ALS ONE 2020 Research Symposium - Together from afar.
Page 2
ABOUT ALS ONE
ALS ONE is a partnership bringing together world-leading ALS researchers, doctors, and care practitioners focused on finding treatments for ALS and novel approaches to improve care and quality of life for those battling the disease. The unprecedented linking of minds and resources from Massachusetts General Hospital (MGH), University of Massachusetts Medical Center, ALS Therapy Development Institute (ALS TDI), and Compassionate Care ALS (CCALS) is unique for its leadership in efficiency, dedication to innovative research, and commitment to increasing access to care and treatment. ALS ONE's partnership includes: Dr. Merit Cudkowicz, MD, MSc, Chief of Neurology and Director Sean M. Healey & AMG Center for ALS at Mass General Hospital (MGH) and the Julieanne Dorn Professor of Neurology Harvard Medical School; Dr. Robert Brown Jr., MD, DPhil, Chief of Neurology, UMass Medical Center; Dr. Steve Perrin, Ph.D., Chief Executive Officer and Vice Chairman at the ALS Therapy Development Institute; Dr. James Berry, MD, Co-Director of the MGH Neuromuscular Division and of the MGH ALS Unit; Nazem Atassi, MD, Associate Director, Neurological Clinical Research Institute (NCRI), Mass General HospitalAssociate Professor of Neurology, Harvard Medical School; and Ron Hoffman, Founder and Director of Compassionate Care ALS (CCALS).
ALS ONE Founder, Kevin Gosnell (left front), with the ALS ONE partnership team in a planning meeting in 2015. Kevin lost his battle to ALS in August of 2016, just 7 months after the official launch of ALS ONE on January 21, 2016. ALS ONE has continued to grow significantly, raising over 10 million dollars, making measurable and meaningful impacts on our ALS research and care.
LEARN MORE AT: ALSONE.ORG
ALS ONE 2020 Research Symposium - Together from afar.
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YOUR HOSTS & COMMITTEE
ALS ONE SCIENTIFIC & SYMPOSIUM COMMITTEE
James Berry, MD, MPH Associate Professor of Neurology at Harvard Medical School, Chief of the MGH Division of ALS and Motor Neuron Diseases, and Director of the MGH Neurological Clinical Research Institute (NCRI).
Robert Brown, MD, DPhil
Merit Cudkowicz, MD
Steven Perrin, PhD
Chair of Neurology, University of Massachusetts Medical Center, Director of the Day Neuromuscular Research Laboratory at the UMass Medical School.
Chief of Neurology and Director Sean M. Healey & AMG Center for ALS at Mass General Hospital and the Julieanne Dorn Professor of Neurology Harvard Medical School.
Chief Executive Officer and Vice Chairman, ALS Therapy Development Institute (ALS TDI).
OPENING REMARKS DAY 1
OPENING REMARKS DAY 2
Mark Sullivan Global Lead, Salesforce ALS ONE Chairman of the Board
Matthew Hogan CEO, Industrial Packaging ALS ONE Board of Directors and former Chairman of the Board, ALS ONE
ALS ONE 2020 Research Symposium - Together from afar.
SYMPOSIUM LOGISTICS COMMITTEE
Jennifer DiMartino Executive Director ALS ONE
Beverly Smits Program Manager NCRI at MGH
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HOSTS & OPENING BIOGRAPHIES In alphabetical order
James Berry, MD, MPH Associate Professor of Neurology at Harvard Medical School. Chief of the MGH Division of ALS and Motor Neuron Diseases, and Director of the MGH Neurological Clinical Research Institute (NCRI).
Dr. Berry provides care for people with amyotrophic lateral sclerosis (ALS). He is the director of the Massachusetts General Hospital (MGH) multidisciplinary amyotrophic lateral sclerosis (ALS) clinic and Chief of the Division of ALS and Motor Neuron Diseases. He has worked to better serve people with ALS by building the tools and team required to extend the reach of the MGH clinic team beyond the walls of the physical clinic. The ALS House Call program and video televisit program are examples of exciting initiatives at the MGH ALS clinic. Dr. Berry also works as an ALS researcher, designing, overseeing, and carrying out trials at MGH and centers across the country. His work is focused on the identification of markers of ALS in blood and spinal fluid, with an emphasis on markers of abnormal inflammation. He collaborates broadly with researchers around the globe on these biomarker efforts. He is also actively working to develop mobile health and digital phenotyping methods to better understand ALS and hasten the development of new therapies, while reducing the burden of trial participation for people with ALS. Dr. Berry is also the Director of the MGH Neurological Clinical Research Institute (NCRI) and Director of the Mass General Brigham Neurodegenerative Clinical Research Fellowship. He is a member of the Massachusetts ALS Registry team and CDC/ATSDR National ALS Registry and Biorepository Expert Panels. In addition, he has served on the Executive Committee of the NEALS Consortium, a national organization for ALS clinical researchers, where he also leads the Technology in ALS subcommittee..
ALS ONE 2020 Research Symposium - Together from afar.
Robert Brown, MD, DPhil Professor and Chair of Neurology at the University of Massachusetts Medical School. Director of the Day Neuromuscular Research Laboratory at the University of Massachusetts Medical School.
Robert H. Brown Jr., MD, DPhil, is director of the Program in Neurotherapeutics at the University of Massachusetts Medical School. Prior to joining the Medical School in May 2008, Dr. Brown was professor of neurology at Harvard Medical School and a neurologist at Massachusetts General Hospital. He also served as Director of the Day Neuromuscular Laboratory and of the Muscular Dystrophy Association Clinic at Mass General Hospital. Born in Boston, Dr. Brown graduated magna cum laude from Amherst College in 1969 with a degree in biophysics. In 1973 he completed a doctorate of philosophy in neurophysiology at Oxford University and received his medical degree from Harvard Medical School in 1975. Following medical school, Dr. Brown completed his internship in internal medicine at Peter Bent Brigham Hospital and a residence in neurology at Massachusetts General Hospital. From 1980 to 1983, Dr. Brown was a research a fellow in neuroscience at Children's Hospital in Boston. In 1975, Brown joined Harvard Medical School as a clinical fellow and rose to the rank of professor of neurology in 1998. Dr. Brown is an internationally known researcher and clinician leading the quest to cure neurodegenerative and neuromuscular diseases like amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease. Dr. Brown founded the Day Neuromuscular Laboratory in 1984 to investigate neuromuscular diseases and continues his work with the Day Lab today at the University of Massachusetts Medical School. In 1993, a team of researchers led by Dr. Brown discovered the first gene linked to the inherited form of ALS, a protein anti-oxidant known as superoxide dismutase, or SOD1. In addition to his work with ALS, Dr. Brown has also identified disease genes in other inherited neurodegenerative and neuromuscular diseases such as hyperkalemic periodic paralysis, Miyoshi muscular dystrophy. and hereditary sensory neuropathy. Page 5
HOST & OPENING BIOGRAPHIES In alphabetical order
Merit Cudkowicz, MD, MsC Chief of Neurology and Director Sean M. Healey & AMG Center for ALS at Mass General Hospital and the Julieanne Dorn Professor of Neurology Harvard Medical School
Dr. Merit Cudkowicz is the Director of the Sean M. Healey & AMG Center for ALS, Chief of Neurology at Mass General, Director and the Julieanne Dorn Professor of Neurology at Harvard Medical School. Dr. Cudkowicz’s research and clinical activities are dedicated to the study and treatment of people with Amyotrophic Lateral Sclerosis (ALS). Dr. Cudkowicz is one of the founders and past Co-Chairs of the Northeast ALS Consortium (NEALS), a group of over 134 clinical sites performing collaborative clinical trials and research in ALS. She has brought innovations to accelerate the development of treatments for people with ALS, including senior role in first antisense oligonucleotide treatment for a neurological disorder (SOD1 ALS), adaptive trial designs, central IRB - all with goal to bring the best treatments rapidly.
Matthew Hogan CEO, Industrial Packaging Board of Directors and former Board Chair of ALS ONE
Matt Hogan, a member of the Board of Directors, served as Chairman for several years. Mr. Hogan received his B.A. from Holy Cross College, and his Master of Business Administration from Boston University's Graduate School of Management. Mr. Hogan is the CEO of Industrial Packaging Supply, Inc., Treasurer of the Objective Management Group, former Chairman of the Board of the Edward M. Kennedy Community Health Center, and a former member of the Board of Directors of the Massachusetts Manufacturing Extension Partnership (MassMEP).
Dr. Cudkowicz is Principal Investigator of the Clinical Coordination Center for the National Institute of Neurological Disorders and Stroke’s Neurology Network of Excellence in Clinical Trials (NeuroNEXT). Dr. Cudkowicz is launching the first platform trial initiative in ALS, the HEALEY ALS Platform Trial, a program that will greatly accelerate therapy development in ALS. Dr. Cudkowicz received the American Academy of Neurology 2009 Sheila Essay ALS award, the 2017 Forbes Norris Award from the International MND Alliance, the 2017 Pinnacle Award from the Boston Chamber of Commerce and the 2019 Ray Adams American Neurological Association Award. A dedicated educator, Dr. Cudkowicz mentors many young neurologists in clinical investigation of ALS and related neurodegenerative disorders. Dr. Cudkowicz completed her undergraduate degree in chemical engineering at Massachusetts Institute of Technology and obtained a medical degree in the Health Science and Technology program of Harvard Medical School. She served her neurology residency and fellowship at MGH.
ALS ONE 2020 Research Symposium - Together from afar.
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HOST & OPENING BIOGRAPHIES In alphabetical order
Steven Perrin, PhD
Mark Sullivan
Chief Executive Officer and Vice Chairman,
Chairman of the Board, ALS ONE
ALS Therapy Development Institute
Strategy & Ecosystems Global Lead - Consumer Goods Industry at Salesforce
Dr. Steven Perrin is the Chief Executive Officer and Vice Chairman at the ALS Therapy Development Institute (ALS TDI) in Cambridge, MA. Dr. Perrin moved into the pharmaceutical industry in 1997, holding positions at the Hoechst-Ariad Genomics Center, Aventis Pharmaceuticals, and more recently at Biogen where he served as Director of Molecular Profiling. Dr. Perrin joined the ALS Therapy Development Institute in 2007 as part of their historical collaboration with the Muscular Dystrophy Association initiative, Augie’s Quest, to develop effective therapeutics for people with ALS. He spearheaded the development of computational biology capabilities and information management systems to more clearly understand the molecular mechanisms associated with disease onset and progression in neurodegenerative diseases. He also helped expand the drug screening program to include expertise in the generation and assessment of gene therapy vectors and protein biologics in preclinical models of neurodegeneration. In 2012, Dr. Perrin led his team in outlining the world’s first Precision Medicine Program (PMP) in ALS, which opened for enrollment in 2014. The PMP aims to characterize and screen potential treatments targeting the various subgroups of ALS by combining progression data collected from patients, using full genome sequencing, and creating disease-relevant cell lines from samples collected from participants. By rapidly deploying funds directly received from the ALS Ice Bucket Challenge, more than 250 people have enrolled in the PMP. Committed to transparency and making people with ALS real partners in research, Dr. Perrin created an online portal through which each enrollee can see their data evolve as it moves through the process. Furthermore, Dr. Perrin has developed a business plan to facilitate pharmaceutical partnerships for rapid clinical development and commercialization of promising therapeutics identified at ALS TDI.
Mark Sullivan is currently the Strategy & Ecosystems Global Lead for the Consumer Goods Industry at Salesforce, joining recently after two years as the Global Business Lead for Banking & Capital Markets at Genpact, where he was responsible for leading growth strategy and execution by developing disruptive solutions for banking clients, as well as managing the continuing evolution of the Banking & Capital Markets’ priority service lines: Financial Crimes, Risk & Analytics, Auto Finance, Transformation Services and Consumer Banking. Prior to joining Genpact, Mark served as the North America leader for IBM’s Banking and Financial Markets Global Business Services and managed some of the company's largest clients. He spent seven years at Accenture, where he assisted with the launch of their Digital Practice and held a leadership role within North America Financial Services Consulting. Other leadership roles have included Chief Operating Officer for Fidelity Investments and Partner at KPMG Consulting. Mark earned a bachelor's degree in economics from Framingham State College and attended graduate school at the University of Hartford.
Dr. Perrin received his doctoral degree from Boston University, studying the transcriptional regulation of genes during adipocyte and myocyte differentiation.
ALS ONE 2020 Research Symposium - Together from afar.
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AGENDA Day 1: November 4th 10:30 – 10:45 AM
Welcome & Introductions
10:45 – 12:45 AM
From SALS to FALS: New biology and therapeutic opportunities. Stathmin-2: an emerging therapeutic target in TDP-43 proteinopathies. Presented by: Kevin Eggan, PhD; Harvard Stem Cell Institute and Clotilde Lagier-Tourenne, MD, PhD; Mass General Hospital. Expanding mechanisms and therapeutic targets for ALS. Presented by: Aaron Gitler, PhD; Stanford Medicine. Astrocyte Hemichannels as Therapeutic Targets for ALS. Presented by: Nicholas Maragakis, MD; Johns Hopkins. Microglia in the inflamed CNS: New targets for therapeutic intervention. Presented by: Dori Schafer, PhD; University of Massachusetts Medical School.
12:45 – 1:15 PM
Break
1:15 – 2:15 PM
2020 View of ALS Therapeutics
2:15 – 3:15 PM
New insights into axonal trafficking and MNs
Presented by: Merit Cudkowicz, MD, MSc; Sean M. Healey & AMG Center, Mass General Hospital and Jeremy Shefner, MD, PhD; Barrow Neurological Institute.
· ALS mechanisms at the crossroads of lysosomal and RNA biology. Presented by: Michael Ward, MD, PhD; NINDS. How to keep a neuron healthy: the role of autophagy and mitophagy in neuronal homeostasis and neurodegeneration. Presented by: Erika Holzbaur, PhD; University of Pennsylvania Perelman School of Medicine.
ALS ONE 2020 Research Symposium - Together from afar.
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AGENDA Day 2: November 5th 10:30 – 10:45 AM
Welcome & Introductions
10:45 – 12:15 AM
Industry Updates Verge Genomics Presented by: Irene Choi, PhD Neuropore Therapies Presented by: Doug Bonhaus, PhD BrainStorm Cell Therapeutics Presented by: Namita Goyal, MD Alexion Pharmaceuticals Presented by: Glen Frick, MD, PhD Biogen Presented by: Thos Cochrane MD, MBA · ProMIS Neurosciences Presented by: Neil R Cashman, MD
12:15 – 1:00 PM
Break
1:00 – 2:15 PM
Industry Updates · Anavex Life Sciences Presented by: Nell Rebowe, BS Prilenia Therapeutics Presented by: Michael Hayden, CM OBC MB ChB PhD FRCP(C) FRSC Mitochon Pharmaceuticals Presented by: John G. Geisler, PhD NeuroSense Therapeutics Presented by: Shiran Zimri, PhD Amylyx Pharmaceuticals Presented by: Joshua Cohen, BS, & Justin Klee, BS
2:15 – 3:45 PM
Continued on next page.
ALS ONE 2020 Research Symposium - Together from afar.
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AGENDA Day 2: November 5th Continued
2:15 – 3:45 PM
HEALEY ALS Platform Trial Round Table Discussion Presented by (in alphabetical order) Marianne Chase, BA; Sean M. Healey & AMG Center, Mass General Hospital Merit Cudkowicz, MD, MSc; Sean M. Healey & AMG Center, Mass General Hospital Eric Macklin, PhD; Mass General Hospital Sabrina Paganoni, MD, PhD; Sean M. Healey & AMG Center, Mass General Hospital Melanie Quintana, PhD; Berry Consultants Irfan Qureshi, MD; Biohaven Pharmaceuticals Jeremy Shefner, MD, PhD; Barrow Neurological Institute
ALS ONE 2020 Research Symposium - Together from afar.
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In alphabetical order
KEYNOTE SPEAKERS
Bio's on following pages
MERIT CUDKOWICZ, MD, MSC Chief of Neurology and Director Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH)) and the Julieanne Dorn Professor of Neurology Harvard Medical School.
KEVIN EGGAN, PHD Professor In The Department of Stem Cell and Regenerative Biology at Harvard University, Director of Stem Cell Biology For The Stanley Center For Psychiatric Research at The Broad Institute.
AARON GITLER, PHD Basic Science Professor Genetics, Stanford Medicine.
ERIKA HOLZBAUR, PHD William Maul Measey Professor Of Physiology, University of Pennsylvania Perelman School Of Medicine.
CLOTILDE LAGIER-TOURENNE, MD, PHD Associate Professor Of Neurology, Harvard Medical School, Associate In Neuroscience, Massachusetts General Hospital, Healey Family ALS Endowed Chair For Research. .
NICHOLAS MARAGAKIS, M.D Medical Director, Johns Hopkins ALS Clinical Trials Unit, Director of the ALS Center for Cell Therapy and Regeneration Research and Professor of Neurology, Johns Hopkins University.
DOROTHY (DORI) SCHAFER, PHD Assistant Professor in Neurobiology, University of Massachusetts Medical School.
JEREMY SHEFNER, MD, PHD Kemper And Ethel Marley Chair of Neurology and Associate Director for Barrow Neurological Institute.
MICHAEL WARD, MD, PHD Principal Investigator, Inherited Neurodegenerative Diseases Unit, NINDS.
ALS ONE 2020 Research Symposium - Together from afar.
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KEYNOTE SPEAKER BIOGRAPHIES In alphabetical order
Merit Cudkowicz, MD, MsC Chief of Neurology and Director Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) and the Julieanne Dorn Professor of Neurology Harvard Medical School
Dr. Merit Cudkowicz is the Director of the Sean M. Healey & AMG Center for ALS, Chief of Neurology at Massachusetts General Hospital (MGH), Director and the Julieanne Dorn Professor of Neurology at Harvard Medical School. Dr. Cudkowicz’s research and clinical activities are dedicated to the study and treatment of people with Amyotrophic Lateral Sclerosis (ALS). Dr. Cudkowicz is one of the founders and past Co-Chairs of the Northeast ALS Consortium (NEALS), a group of over 134 clinical sites performing collaborative clinical trials and research in ALS. She has brought innovations to accelerate the development of treatments for people with ALS, including senior role in first antisense oligonucleotide treatment for a neurological disorder (SOD1 ALS), adaptive trial designs, central IRB - all with goal to bring the best treatments rapidly. Dr. Cudkowicz is Principal Investigator of the Clinical Coordination Center for the National Institute of Neurological Disorders and Stroke’s Neurology Network of Excellence in Clinical Trials (NeuroNEXT). Dr. Cudkowicz is launching the first platform trial initiative in ALS, the HEALEY ALS Platform Trial, a program that will greatly accelerate therapy development in ALS. Dr. Cudkowicz received the American Academy of Neurology 2009 Sheila Essay ALS award, the 2017 Forbes Norris Award from the International MND Alliance, the 2017 Pinnacle Award from the Boston Chamber of Commerce and the 2019 Ray Adams American Neurological Association Award. A dedicated educator, Dr. Cudkowicz mentors many young neurologists in clinical investigation of ALS and related neurodegenerative disorders. Dr. Cudkowicz completed her undergraduate degree in chemical engineering at Massachusetts Institute of Technology and obtained a medical degree in the Health Science and Technology program of Harvard Medical School. She served her neurology residency and fellowship at MGH.
ALS ONE 2020 Research Symposium - Together from afar.
Kevin Eggan, PhD Professor In The Department of Stem Cell and Regenerative Biology at Harvard University Director Of Stem Cell Biology For The Stanley Center For Psychiatric Research at The Broad Institute
For the past 16 years, Kevin Eggan has been a Professor in the Department of Stem Cell and Regenerative Biology at Harvard University, a principal investigator at the Harvard Stem Cell Institute and an Institute member at the Broad Institute’s Stanley Center for Psychiatric Research. As a young investigator in the burgeoning field of stem cell biology, Dr. Eggan garnered international recognition for his seminal work and a number of high profile awards for his creativity and productivity, including the MacArthur Foundation “Genius Grant” in 2006. In 2009, he was selected as one of 50 Howard Hughes Medical Institute (HHMI) Early Career Scientists who received six years of dedicated support to conduct transformative research. His current research applies stem cell technologies to studying the mechanisms through which genetic variants confers risk for psychiatric and neurodegenerative diseases. In 1996, Dr. Eggan received his bachelor’s degree in microbiology from the University of Illinois. He completed a two-year predoctoral fellowship at the National Institutes of Health, before pursuing his doctoral studies in biology at The Massachusetts Institute of Technology. There, he actively pursued projects focused on cloning, stem cells, and reprogramming after nuclear transfer under the guidance of genetics pioneer Rudolf Jaenisch. After completing his doctoral studies in 2002, Eggan stayed in Jaenisch’s lab for one year of postdoctoral collaboration with Richard Axel from Columbia University. In 2003, he moved to Harvard University as an independent Junior Fellow and then became an Assistant Professor of Molecular and Cellular Biology in 2005. In 2013, he was promoted to the Rank of Full Professor in The Department of Stem Cell and Regenerative Biology, the first Harvard academic department to span the Medical School and School of Arts and Sciences.
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KEYNOTE SPEAKER BIOGRAPHIES In alphabetical order
Aaron Gitler, PhD Professor
Erika Holzbaur, PhD William Maul Measey Professor Of Physiology,
Stanford Medicine University Of Pennsylvania Perelman School Of Medicine
Aaron Gitler is the Stanford Medicine Basic Science Professor in the Department of Genetics at Stanford University. He received his B.S. degree from Penn State University and did his PhD studies on cardiovascular development in the laboratory of Dr. Jonathan Epstein at the University of Pennsylvania. Then he performed his postdoctoral training with Dr. Susan Lindquist at the Whitehead Institute for Biomedical Research and MIT. In 2007, he established his laboratory at the University of Pennsylvania and moved to Stanford in 2012. His laboratory has been using a combination of yeast and human genetics approaches to investigate pathogenic mechanisms of ALS.
ALS ONE 2020 Research Symposium - Together from afar.
Erika L. F. Holzbaur is the William Maul Measey Professor of Physiology at the University of Pennsylvania Perelman School of Medicine. She received her B.S. in Chemistry and History from the College of William and Mary, her PhD in Biochemistry from Penn State, and performed her postdoctoral studies at the Worcester Foundation for Experimental Biology. Dr. Holzbaur was recruited to Penn in 1992 and now holds an endowed chair. The Holzbaur lab studies the dynamics of organelle motility along the cellular cytoskeleton, required to drive the active transport of organelles along the axons and dendrites of neurons. The lab also investigates cellular mechanisms leading to neurodegeneration in diseases such as ALS and Parkinson’s, focusing on autophagy and mitophagy, pathways critical to maintain neuronal homeostasis. Dr. Holzbaur was appointed a Porter Fellow and has received the NINDS Javits Award, the Stanley N. Cohen Biomedical Research Award, and is an inaugural Lifetime Fellow of the American Society for Cell Biology.
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KEYNOTE SPEAKER BIOGRAPHIES In alphabetical order
Clotilde LagierTourenne, MD, PhD Associate Professor Of Neurology, Harvard Medical School Associate In Neuroscience, Massachusetts General Hospital Healey Family ALS Endowed Chair For Research Dr. Clotilde Lagier-Tourenne investigates the molecular mechanisms driving neurodegeneration in amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and Huntington’s disease. Mutations and/or cellular mislocalization of several RNA binding proteins have been identified as central components in the pathogenesis of ALS and FTD. Using innovative cellular and genomics techniques, her group explores the regulatory networks between RNA binding proteins and changes in RNA expression that occur in these diseases. The team also develops cellular and animal models to uncover mechanistic insights underlying neuronal death in ALS and FTD patients with C9ORF72 expansion, the most common genetic cause of these conditions. Clotilde has established collaborations with academic and pharmaceutical partners to develop novel approaches to therapy, including RNA-targeting antisense oligonucleotides and immunotherapies for patients with ALS and FTD. Clotilde Lagier-Tourenne is Associate Professor of Neurology at the Massachusetts General Hospital and Harvard Medical School. She is a member of the Sean M. Healey & AMG Center for ALS at Mass General and an associate member at the Broad Institute. She trained as a medical geneticist in France and at Columbia University. After a postdoctoral fellowship with Dr. Don Cleveland, she became Assistant Professor at UCSD in 2013, and moved to the Massachusetts General Hospital in 2015. She received the Alphonse Laveran Prize, the Milton-Safenowitz Fellowship, the MDA Career Development and Frick Foundation Awards, the 6th International Paulo Gontijo Award in Medicine, the 4th annual Jenny S. Henkel Lectureship, the 2019 Lisa S. Krivickas Lectureship, and the 2017 Grass Foundation – American Neurological Association Award in Neuroscience.
ALS ONE 2020 Research Symposium - Together from afar.
Nicholas Maragakis, M.D. Medical Director Johns Hopkins ALS Clinical Trials Unit, Director of the ALS Center for Cell Therapy and Regeneration Research and Professor of Neurology Johns Hopkins University
Dr. Maragakis is the Medical Director of the Johns Hopkins ALS Clinical Trials Unit and Director of the ALS Center for Cell Therapy and Regeneration Research and Professor of Neurology at Johns Hopkins University. The ALS Clinic at John Hopkins is a world recognized leader in providing medical car and offering the latest in clinical trials and therapies to ALS patients. A common theme to his research has been in the study of the astrocyte biology and its role in disease pathogenesis with a particular emphasis in how astrocytes may contribute to Amyotrophic Lateral Sclerosis (ALS). His laboratory in collaboration with others, has been interested in the development of induced pluripotent stem cells from ALS patients. His laboratory has created over 50 iPS cell lines from familial and sporadic ALS patients as well as controls. His current efforts have been to characterize iPSC-derived motor neurons and astrocytes both in vitro and in vivo with an effort towards understanding disease mechanisms; in particular modeling ALS disease heterogeneity with regard to disease progression. Dr. Maragakis has also been heavily involved in clinical research as the principal investigator, site principal investigator, or co-investigator of numerous clinical trials in ALS, many coordinated by the Northeast ALS Consortium on which he has served as an Executive Board member and currently a member of the Scientific Advisory Board. Dr. Maragakis has received several awards for his outstanding research efforts and received the Certification of Meaningful Use Stage 1 EHR from the Centers for Medicare& Medicaid Services (CMS) in 2013. He is member of the American Board of Psychiatry and Neurology, the American Academy of Neurology (AAN) and the Northeast ALS Consortium (NEALS). Dr. Maragakis received his Bachelors of Science degree and medical degree from the University of Utah and he completed his residency and fellowship in neurology at John Hopkins University.
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KEYNOTE SPEAKER BIOGRAPHIES In alphabetical order
Dorothy (Dori) Schafer, PhD Assistant Professor in Neurobiology University of Massachusetts Medical School
Dorothy (Dori) Schafer received her bachelor’s degree in Neuroscience from Mount Holyoke College in 2001 and her PhD in Biomedical Science from the University of Connecticut Health Center in 2008. She then began her postdoctoral training at Boston Children’s Hospital/Harvard Medical School in Dr. Beth Stevens’ laboratory. Here, she made the discovery that microglia, the resident CNS macrophages, sculpt neural circuits in the developing brain by engulfing a subset of less active synapses. Dr. Schafer is considered a leader in the field studying microglial function within neural circuits. Her earlier postdoctoral work showing microglia engulf and eliminate synapses via the classical complement cascade has served as a foundation for the field to understand new roles for microglia in regulating synaptic connectivity in health and disease. Dr. Schafer joined UMass Medical School in 2015 as an Assistant Professor where she was a recipient of a NIH R00 Pathway to Independence Award (NIMH), Charles H. Hood Child Research Award, a NARSAD Young Investigator Award, and the Brain Behavior Research Foundation Freedman Award Honorable Mention. Her laboratory uses a combination of cutting-edge molecular genetic approaches and imaging to uncover novel roles for microglia in modulating neural circuit structure and function in health and in neuroinflammatory diseases of the CNS.
ALS ONE 2020 Research Symposium - Together from afar.
Jeremy Shefner, MD, PhD Kemper And Ethel Marley Chair Of Neurology And Associate Directovr Barrow Neurological Institute
Dr. Jeremy Shefner, MD, PhD is Chair of the Department of Neurology, Professor of Neurology, and Senior Vice President of Barrow Neurological Institute at Dignity Health St. Joseph’s Hospital and Medical Center in Phoenix. He also serves oN the Healey Center Scientific Advisory Council. Dr. Shefner received his PhD in sensory physiology from the University of Illinois in Urbana-Champaign, and his MD from Northwestern University Medical School in Chicago. He completed his residency training at the Harvard Longwood Neurology Training Program in Boston and completed a fellowship in neuromuscular disease at the Brigham and Women’s Hospital in Boston. Dr. Shefner’s research focuses on biomarker development and the clinical therapeutics of ALS and spinal muscular atrophy. He is one of the cofounders of the Northeast ALS Clinical Trials Consortium (NEALS), the largest and most active consortium in the world dedicated to ALS. He continues to direct the NEALS outcomes and clinical monitoring cores and is currently the principal investigator for two multicenter clinical trials, as well as a biomarker study evaluating the effectiveness of electrical impedance myography in measuring ALS disease progression. Dr. Shefner has published approximately 200 papers in peer reviewed journals and has served on multiple grant review panels. He has also participated in committees organized by the Institute of Medicine to investigate the relationship between military service and ALS, as well as the health effects of Agent Orange on Vietnam War veterans. Dr. Shefner received the Sheila Essey Award for ALS Research, presented annually by the American Academy of Neurology and the ALS Association (2014).
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KEYNOTE SPEAKER BIOGRAPHIES In alphabetical order
Michael Ward, MD, PhD Principal Investigator, Inherited Neurodegenerative Diseases Unit NINDS
Dr. Ward received his B.S. from Kenyon College in 1999 and M.D. and Ph.D. degrees from Washington University in St. Louis in 2007. As a graduate student, he worked in Yi Rao’s lab and studied the regulation of cell migration during neurodevelopment. Following a neurology residency at the University of California in San Francisco, he sub-specialized in behavioral neurology and completed a postdoctoral fellowship in Li Gan’s lab studying basic mechanisms of frontotemporal dementia. As a fellow he received an American Brain Foundation CRTF award and a NIH K08 career development award. In 2015 he joined the NINDS as an Assistant Clinical Investigator, and became an Investigator in 2017. In addition to leading a research team, he sees patients with ALS/FTD spectrum disorders in the NIH Neurodegeneration Clinic. Since 2019, he has co-directed the iPSC Neurodegenerative Disease Initiative, a large multicenter project tasked with creating and phenotyping hundreds of isogenic iPSC models of Alzheimer’s Disease and related dementias. His current research focuses on identifying intersecting molecular mechanisms of neurodegenerative diseases, with an ultimate goal of developing targeted, disease-modifying therapies for affected patients.
ALS ONE 2020 Research Symposium - Together from afar.
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In alphabetical order
INDUSTRY SPEAKERS
Bio's on following pages
DOUGLAS BONHAUS,PH.D. CEO & CSO, Neuropore Therapies, Inc
NEIL CASHMAN, MD Principal Investigator, Inherited Neurodegenerative Diseases Unit at NINDS
IRENE CHOI, PHD Director of Drug Discovery at Verge Genomics
THOS. COCHRANE MD, MBA Global Medical Lead, ALS, Biogen, Inc.
JOSHUA COHEN, BS Co-Founder and Co-CEO, Amylyx Pharmaceuticals
GLEN FRICK, MD, PHD Executive Medical Director, Alexion Pharmaceuticals
JOHN G. GEISLER, PHD Founder & Chief Scientific Officer at Mitochon Pharmaceuticals, Inc.
NAMITA GOYAL, MD Associate Professor of Neurology, Director of the ALS clinic, Director of the Neuromuscular Medicine Fellowship program, and Director of the Neurology Clinical Trial Unit at the Univ. of California, Irvine.
MICHAEL HAYDEN, CM, OBC, MB,CHB, PHD, FRCP(C), FRSC Co-Founder & CEO, Prilenia Therapeutics
JUSTIN KLEE, BS Co-Founder and Co-CEO, Amylyx Pharmaceuticals
NELL REBOWE, BS Director of Business Development, Anavex Life Sciences Corp.
SHIRAN ZIMRI, PHD Project Manager, Parkinson’s lead at NeuroSense Therapeutics
ALS ONE 2020 Research Symposium - Together from afar.
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INDUSTRY SPEAKER BIOGRAPHIES In alphabetical order
Douglas Bonhaus, Ph.D. CEO & CSO Neuropore Therapies, Inc
Neil Cashman, MD Principal Investigator, Inherited Neurodegenerative Diseases Unit NINDS
Dr. Douglas Bonhaus is a drug-discovery scientist with more than twenty years of experience in the pharmaceutical industry. Initially trained in pharmacology and toxicology at the University of Arizona, Dr. Bonhaus has held leadership positions at Syntex, Roche and ACADIA Pharmaceuticals. Dr. Bonhaus was also cofounder of Denysias Bioscience, a company focused on developing a novel therapeutic for impulse control disorders in patients with Parkinson’s disease. Scientific and leadership efforts in these positions have contributed to the advancement of a number of novel therapeutic agents into clinical development. Dr. Bonhaus now serves as the President and Chief Executive Officer of Neuropore Therapies.
ALS ONE 2020 Research Symposium - Together from afar.
Dr. Neil Cashman is co-founder and CSO of ProMIS Neurosciences, a Canadian biotech company focused on therapeutics and diagnostics for neurodegenerative diseases. He is also a Professor of Medicine at the University of British Columbia, where his research lab is focused on the role of protein misfolding in neurodegenerative diseases, particularly ALS. He is a Director of the UBC ALS Centre Clinic at G.F. Strong Hospital.
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INDUSTRY SPEAKER BIOGRAPHIES In alphabetical order
Irene Choi, PhD
Thos. Cochrane MD, MBA
Director of Drug Discovery
Global Medical Lead, ALS
Verge Genomics
Biogen, Inc.
Irene is the Director of Drug Discovery at Verge Genomics in San Francisco, CA. She has spent her career in early drug discovery in neuroscience and oncology having led multiple programs, one of which was of a novel analgesic with demonstrated efficacy in Phase 3 clinical trials. At Verge Genomics, she works with biologists and computational scientists to test machine learning predictions at the bench to identify novel therapeutics in neurodegenerative diseases.
ALS ONE 2020 Research Symposium - Together from afar.
Dr. Cochrane is the Global Medical Lead for ALS at Biogen. He is an adult neuromuscular neurologist who worked at Brigham and Women’s Hospital from 2004-2017, where he specialized in neuromuscular and electrodiagnostic medicine. He also specialized in medical ethics, and was the first Director of Neuroethics at the Center for Bioethics at Harvard Medical School.
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INDUSTRY SPEAKER BIOGRAPHIES In alphabetical order
Joshua Cohen, BS
Glen S. Frick, MD, PhD
Co-Founder and Co-CEO
Executive Medical Director
Amylyx Pharmaceuticals
Alexion Pharmaceuticals
Joshua Cohen co-founded Amylyx Pharmaceuticals in 2013 and continues to serve as its co-CEO. Josh has overseen the growth of Amylyx from a dorm-room concept to a clinical stage pharmaceutical company. His research experience includes work both at Brown University and at the National Institute of Standards in Technology. Josh received his BS in biomedical engineering from Brown University.
ALS ONE 2020 Research Symposium - Together from afar.
Glen S. Frick, MD, PhD is an NIH FellowshipTrained Pharmacologist and Board-Certified Physician with over fifteen years of extensive Phase 1-4 clinical trial experience. Glen has held key leadership roles within the pharmaceutical industry, including GlaxoSmithKline, Wyeth/Pfizer, Shire and Endo. He has conducted clinical and translational medicine studies in Neurodegeneration, Respiratory, Inflammation, Psychiatry, Pain and Dermatology. Glen completed an MD/PhD program at SUNYDownstate Medical Center obtaining his PhD in Developmental Neural & Behavioral Science. He served as VP of Clinical Development & Operations at Endo Pharmaceuticals where he oversaw all Clinical Development Programs. Glen served as Chief Medical Officer at Clene Nanomedicine, a startup biotech company in Salt Lake City, UT where he led the strategization and implementation of clinical development programs focusing on several Neurodegeneration and Neuroinflammation indications. Prior to joining Alexion as an Executive Medical Director in Neurology, Glen was a Neurology Global Clinical Trial Leader at Covance where he oversaw various phase clinical trials in a number of pediatric and rare disease neurology indications including gene therapy studies.
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INDUSTRY SPEAKER BIOGRAPHIES In alphabetical order
John G. Geisler, PhD
Namita Goyal, MD
Founder & Chief Scientific Officer
Associate Professor of Neurology
Mitochon Pharmaceuticals, Inc.
Director of the ALS clinic, Director of the Neuromuscular Medicine Fellowship program, and Director of the Neurology Clinical Trial Unit at the University of California, Irvine
Expertise in drug discovery in metabolic disease (Pfizer, Ionis, JnJ) with a primary role bringing forward the inception of new ideas. By striving to working at the root of problems and years of attempting to partition lipids to restore insulin sensitivity, Dr. Geisler has built an expertise in mitochondrial energy expenditure. From this work, stemmed a broader understanding of new mitochondrial targeted applications, and significant gaps of pharmacological interventions for insidious movement disorders, neurodegenerative diseases, and trauma.
Dr. Namita Goyal is an Associate Professor of Neurology at University of California, Irvine, and specializes in Neuromuscular Medicine. She serves as the Director of the ALS clinic, Director of the Neuromuscular Medicine Fellowship program, and Director of the Neurology Clinical Trial Unit at UC Irvine. Dr. Goyal has authored several neuromuscular articles and has given many national talks on neuromuscular diseases. In addition to evaluating and treating patients, she is a lead site investigator in several clinical trials involving neuromuscular diseases, with a special focus on ALS and myositis.
Dr. Geisler holds a Ph.D. in Mammalian Genetics from University of Tennessee, conducted at Oak Ridge National Laboratories (ORNL), and a dual fellowship at Pfizer & Yale University, focused on the physiology of Type-2 diabetes and whole-body flux. Mitochon Pharmaceuticals was launched Nov 2014 along with co-founder, Robert Alonso and currently in Phase I to test the safety and tolerability of MP-101 (2, 4 dinitrophenol) in NHV. With successful completion, Mitochon is planning for a series of Phase IIa biomarker studies to test the merits of MP-101 to lower damage caused by mitochondria dysfunction and increase biogenesis/neurotrophins for repair in ALS, Huntington’s and Alzheimer’s Disease participants.
ALS ONE 2020 Research Symposium - Together from afar.
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INDUSTRY SPEAKER BIOGRAPHIES In alphabetical order
Michael Hayden, CM OBC MB ChB PhD FRCP(C) FRSC
Justin Klee, BS
Co-Founder & CEO
Co-Founder and Co-CEO
Prilenia Therapeutics
Amylyx Pharmaceuticals
Dr. Hayden is an accomplished scientist and physician. He is a Killam Professor at the University of British Columbia. Dr. Hayden is also a Canadian Research Chair in Human Genetics and Molecular Medicine. He was the President of Global R&D and Chief Scientific Officer at Teva from 2012-2017. During this time approximately 35 new products were approved mainly for diseases of the CNS. In 2017 Teva R&D ranked top of the industry for CNS development and clinical trial success.
Justin Klee co-founded Amylyx Pharmaceuticals in 2013 and continues to serve as co-CEO. Along with his co-founder Josh Cohen, Justin has overseen the development of Amylyx’s novel platform and therapeutic for neurodegenerative diseases from initial testing through clinical trials. Justin previously did research at Brown University in neural systems and neurophysiology and Alzheimer’s disease at Harvard Medical School. He received his Sc.B in Neuroscience from Brown University.
Dr. Hayden was named one of the 50 Canadians born in the 20th century who have changed the world. Dr. Hayden is the co-founder of five biotechnology companies including: Prilenia, NeuroVir Therapeutics Inc., Xenon Pharma-ceuticals Inc., Aspreva Pharmaceuticals Corp and 89bio. Author of approximately 900 peer-reviewed publications, Dr. Hayden has focused his research primarily on translational medicine, including neurodegenerative disorders such as Huntington disease and ALS, predictive, personalized medicine and drug development. Dr. Hayden was inducted into the Canadian Medical Hall of Fame in 2017. He was named one of PharmaVoice’s “100 of the Most Inspiring People” (2015); awarded an Honorary Doctorate of Science from the University of Alberta (2009) and by the University of Gottingen (2014); the Diamond Jubilee Medal (2012), on behalf of HRH Queen Elisabeth II, in recognition of his signif-icant contributions and achievements; the Killam Prize by the Canada Council of the Arts (2011), in recognition of his outstanding career achievements; and the Canada Gairdner Wightman award also in 2011. Dr. Hayden has also been awarded the Order of Canada (2011), and the Order of British Columbia (2010). He was named Canada’s Health Researcher of the Year by CIHR (NIH of Canada) in 2008, and he received the Canadian Prix Galien in 2007. Dr. Hayden is committed to empowering others. In addition to mentoring over 100 graduate students and postdocs, he is also a TED mentor.
ALS ONE 2020 Research Symposium - Together from afar.
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INDUSTRY SPEAKER BIOGRAPHIES In alphabetical order
Nell Rebowe, BS
Shiran Zimri, PhD
Director of Business Development
Project Manager, Parkinson’s lead
Anavex Life Sciences Corp.
NeuroSense Therapeutics
Nell Rebowe helped spearhead Anavex’s initiative in developing Sigma-1 Modulators and is excited to share the Company’s preclinical data and rationale for Sigma-1 receptors for the treatment of ALS. A Louisiana native, she graduated with a degree in neuroscience from Tulane University. She has lived all over the world and calls New York City home.
Dr. Zimri has over 10 years of experience in neuroscience, researching neurodegenerative diseases, specifically Alzheimer’s disease. She has published many articles in major journals and has been awarded over 12 awards and scholarships for her academic achievements. She has presented her work in numerous national and international venues. Shiran is a certified CRA and holds a PhD in Neurobiology, from Tel-Aviv University.
ALS ONE 2020 Research Symposium - Together from afar.
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In alphabetical order
ROUNDTABLE PANELISTS
Bio's on following pages
MARIANNE CHASE, BA Senior Director Clinical Trial Operations, Neurological Clinical Research Institute at Massachusetts General Hospital
MERIT CUDKOWICZ, MD, MSC Chief of Neurology and Director Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH)) and the Julieanne Dorn Professor of Neurology Harvard Medical School. (Bio located in Keynote Speaker Profiles, on page 12)
ERIC MACKLIN, PHD Biostatistician, MGH Biostatistics Center. Massachusetts General Hospital
SABRINA PAGANONI, MD, PHD Co-Director of the Neurological Research Institute at Massachusetts General Hospital; Assistant Professor at Harvard Medical School.
MELANIE QUINTANA, PHD Senior Statistical Scientist at Berry Consultants
IRFAN QURESHI, MD Vice President, Neurology at Biohaven Pharmaceuticals.
JEREMY SHEFNER, MD, PHD Kemper And Ethel Marley Chair of Neurology and Associate Director For Barrow Neurological Institute (Bio located in Keynote Speaker Profiles, on page 15)
ALS ONE 2020 Research Symposium - Together from afar.
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ROUNDTABLE PANELIST BIOGRAPHIES In alphabetical order
Marianne Chase, BA Senior Director Clinical Trial Operations, Neurological Clinical Research Institute.
Eric Macklin, PhD Biostatistician MGH Biostatistics Center. Massachusetts General Hospital
Massachusetts General Hospital
Marianne Kearney Chase is currently the Senior Director of Clinical Trial Operations for the Neurological Clinical Research Institute (NCRI) and Healey Center for ALS at MGH. The Healey Center, along with key collaborators, designed and initiated the first Platform Trial for Amyotrophic Lateral Sclerosis, which launched in 2020. The NCRI is the Clinical Coordinating Center for the National Institute of Neurological Diseases and Stroke (NINDS) Network for Excellence in Neuroscience Clinical Trials (NeuroNEXT); the Clinical and Data Coordinating Center for the Northeast Amyotrophic Lateral Sclerosis (NEALS) Network and the Parkinson’s Study Group (PSG) Network. Marianne is an active member of the Clinical Trial Transformation Initiative (CTTI) and has over 25 years’ experience in both investigator-initiated NIH / foundation funded and industry sponsored research including Pre-Clinical Study Design, Clinical Study Coordination, Clinical Site Management, Protocol Development, Regulatory Compliance and Project Management. In addition, serves as an adjunct faculty member for the NINDS funded Clinical Trials Methodology Course and has developed and presented courses on various topics including; Good Clinical Practice, Good Source Documentation and Data Management Practices, Adverse Event Reporting, Standard Operating Procedures (SOPs), Overall Management of Multi-Center Clinical Trials, and Complexity of Clinical Trial Design: Ethical and Practical Challenges.
ALS ONE 2020 Research Symposium - Together from afar.
Eric Macklin is a biostatistician in the Biostatistics Center of Massachusetts General Hospital. His work is focused on clinical trials of neurodegenerative and neurodevelopmental diseases. He was an organizing member of the Airlie House ALS Clinical Trials Guidelines writing committee and led the biostatistics group. He provided biostatistical support for the trials of Nuedexta and mexiletine that indicated symptomatic benefit for bulbar dysfunction and muscle cramps. He assisted with the first-inhuman trial of antisense oligonucleotide therapy for SOD1 ALS and the recent CENTAUR trial of sodium phenylbutyrate and taurursodiol. In addition to his support of the HEALEY ALS Platform trial, he is currently the lead statistician for four other ALS trials, previously supported to eight other ALS trials, and regularly serves as an instructor for the Clinical Research Learning Institute for ALS ambassadors. Outside of ALS research, he is actively engaged in trials in Parkinson disease, autism, and Down syndrome and application of mind-body therapies.
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ROUNDTABLE PANELIST BIOGRAPHIES Sabrina Paganoni, MD, PhD Co-Director of the Neurological Research Institute at Massachusetts General Hospital.
Melanie Quintana, PhD Senior Statistical Scientist Berry Consultants
Assistant Professor at Harvard Medical School.
Sabrina Paganoni, MD, PhD is a physician investigator at the Sean M. Healey & AMG Center for ALS at Mass General, Co-Director of the Neurological Research Institute at the Massachusetts General Hospital and Assistant Professor at Harvard Medical School. Dr Paganoni’s research focuses on clinical trials and therapy development for ALS. She is the coPrincipal Investigator of the HEALEY ALS Platform Trial, the first platform trial for ALS in the world. She is the Principal Investigator of the CENTAUR trial as well as other ALS projects that leverage novel trial designs to accelerate the development of new interventions for ALS. Her research has been funded by the NIH, nonprofits, and industry; she published more than 90 peer-reviewed manuscripts. Dr. Paganoni is also passionate about clinical care innovation and access to research for people with ALS. She co-chairs the Upper Motor Neuron Task Force, the Technology Committee and the Recruitment/Retention/Experience Committee at NEALS.
ALS ONE 2020 Research Symposium - Together from afar.
Melanie Quintana is currently a Senior Statistical Scientist at Berry Consultants, where she specializes in designing Bayesian adaptive clinical trials across a wide range of therapeutic areas. Her work includes numerous examples in designing platform trials and clinical trials in rare and progressive disease with a focus on developing models of disease progression to design better and more powerful clinical trials. Before joining Berry Consultants, she earned her Ph.D. in Statistics from Duke University and went on to pursue a Postdoc in Biostatistics at The University of Southern California. While at Duke and USC, she worked closely with collaborators within multiple large studies and consortiums around the nation to develop and implement analytical strategies to assess genetic risk factors for various complex diseases. These collaborations sparked and continue to fuel her interests in developing Bayesian approaches for complex clinical questions.
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ROUNDTABLE PANELIST BIOGRAPHIES Irfan Qureshi, MD Vice President, Neurology Biohaven Pharmaceuticals
Irfan has 17+ years of experience in drug development, clinical and translational research, and patient care. At Biohaven, he serves as Head of Neurodegenerative Disease and is responsible for advancing a portfolio of earlyand late-stage investigational medicines including verdiperstat, an orally administered myeloperoxidase inhibitor that is currently being evaluated in the HEALEY ALS Platform Trial. Previously, at Bristol-Myers Squibb, he was clinical lead for development programs in neurodegenerative, neurodevelopmental, and genetic disease indications utilizing diverse modalities (small molecule, antibody, antisense oligonucleotide, and gene therapy). He joined BMS from the Albert Einstein College of Medicine, where he still holds appointments of Clinical Assistant Professor in the Departments of Neurology and Medicine (Critical Care).
ALS ONE 2020 Research Symposium - Together from afar.
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WITH GRATITUDE TO OUR SPONSORS In alphabetical order
GOLD SPOTLIGHT
ALS ONE 2020 Research Symposium - Together from afar.
With gratitude to Alexion for their generosity in support of our 2020 ALS Research Symposium.
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WITH GRATITUDE TO OUR SPONSORS In alphabetical order
GOLD SPOTLIGHT
ALS ONE 2020 Research Symposium - Together from afar.
With gratitude to Biogen for their generosity in support of our 2020 ALS Research Symposium.
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WITH GRATITUDE TO OUR SPONSORS In alphabetical order
GOLD SPOTLIGHT
ALS ONE 2020 Research Symposium - Together from afar.
With gratitude to BrainStorm for their generosity in support of our 2020 ALS Research Symposium.
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WITH GRATITUDE TO OUR SPONSORS In alphabetical order
GOLD SPOTLIGHT
ALS ONE 2020 Research Symposium - Together from afar.
With gratitude to Cytokinetics for their generosity in support of our 2020 ALS Research Symposium.
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WITH GRATITUDE TO OUR SPONSORS In alphabetical order
BRONZE SPOTLIGHT
ALS ONE 2020 Research Symposium - Together from afar.
With gratitude to Mitsubishi Tanabe Pharma America for their generosity in support of our 2020 ALS Research Symposium.
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WITH GRATITUDE TO OUR SPONSORS In alphabetical order
BRONZE SPOTLIGHT
ALS ONE 2020 Research Symposium - Together from afar.
With gratitude to Biohaven for their generosity in support of our 2020 ALS Research Symposium.
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WITH GRATITUDE TO OUR SPONSORS In alphabetical order
BRONZE SPOTLIGHT
ALS ONE 2020 Research Symposium - Together from afar.
With gratitude to Clene Nanomedicine for their generosity in support of our 2020 ALS Research Symposium.
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NOTES
ALS ONE 2020 Research Symposium - Together from afar.
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NOTES
ALS ONE 2020 Research Symposium - Together from afar.
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DRIVE TO END ALS THE MASSACHUSETTS ALS LICENSE PLATE On September 26, 2016, alongside Governor Charlie Baker, Massachusetts ALS organizations, pALS, and advocates, our license plate campaign was officially launched (see picture below). Just two years later, in June of 2018, the license plates made their debut on cars. The Massachusetts ALS license plate bears the initials of Lou Gehrig, for whom the disease was named, and is available at all full-service RMV's in the state, and by ordering online at ALSplate.org. We hope that in time, the Massachusetts ALS license plate will be as lucrative in helping to end ALS as the first Massachusetts charity plate, the Cape & Islands Plate (raising over $24 million since its debut in 19950, has been in supporting economic growth in that region. We hope if you are a Massachusetts driver, that you'll DRIVE TO END ALS.
ALS ONE 2020 Research Symposium - Together from afar.
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Thank you for joining us from afar for our 2020 ALS Research Symposium.
TOGETHER, WE ARE MAKING THE WORLD A BETTER PLACE. We wish you and your family a happy, healthy and safe holiday season.
From all of us at
With gratitude to this year's Symosium sponsors: